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2018 Medicines in Development for Heart Disease and Stroke
Acute Coronary Syndrome
Drug Name Sponsor Indication Development Status
CER-001 Cerenis Therapeutics reduce risk of cardiovascular events Phase II
(recombinant HDL) Labège, France post-acute coronary syndrome www.cerenis.com
dalcetrapib DalCor Pharmaceuticals acute coronary syndrome Phase III
(CETP inhibitor) San Mateo, CA (genetically-defined population) www.dalcorpharma.com
Praluent® Regeneron acute coronary syndrome Phase III
alirocumab Tarrytown, NY (see also lipid disorders) www.regeneron.com
Sanofi www.sanofi.com
Bridgewater, NJ
Adjunctive Therapies
Drug Name Sponsor Indication Development Status
andexanet alfa Portola Pharmaceuticals anticoagulant reversal application submitted
ORPHAN DRUG South San Francisco, CA (Fxa inhibitor) www.portola.com
ciraparantag Perosphere anticoagulant reversal (Fast Track) Phase II
Danbury, CT www.perospherepharma.com
CMX-2043 Ischemix reduce cardiac injury in patients Phase II completed
(proto-oncogene-protein-c-akt North Grafton, MA undergoing percutaneous coronary www.ischemix.com
modulator) intervention (PCI)
Adjunctive Therapies
Drug Name Sponsor Indication Development Status
Kengreal® Chiesi USA partial or complete obstruction of Phase I
cangrelor Cary, NC systemic to pulmonary artery shunt www.chiesiusa.com
in patients at risk for thrombosis
(neonates)
levosimendan IV Tenax Therapeutics low cardiac output syndrome Phase III
(R-enantiomer of simendan) Morrisville, NC prevention in high risk cardiac surgery www.tenaxthera.com
ORPHAN DRUG (see also pulmonary vascular disease)
Arrhythmias
Drug Name Sponsor Indication Development Status
bucindolol ARCA Biopharma prevention of symptomatic atrial Phase II/III
(beta blocker/vasodilator) Westminster, CO fibrillation/atrial flutter in patients www.arcabio.com
with heart failure (Fast Track)
budiodarone Espero Biopharma atrial fibrillation, ventricular tachycardia Phase II
(calcium, potassium, sodium Jacksonville, FL in patients with implantable www.esperobio.com
channel antagonist) cardioverter defibrillators
etripamil Milestone Pharmaceuticals treatment of symptomatic acute Phase II
(calcium channel antagonist) Quebec, Canada episodes of paroxysmal supraventricular www.milestonepharma.com
tachycardia
flecainide inhalation InCarda Therapeutics paroxysmal atrial fibrillation Phase I
(sodium channel antagonist) Brisbane, CA www.ioncardatherapeutics.com
Arrhythmias
Drug Name Sponsor Indication Development Status
vernakalant IV Cardiome Pharma atrial fibrillation Phase III completed
ORPHAN DRUG Vancouver, Canada www.cardiome.com
Cardiomyopathy
Drug Name Sponsor Indication Development Status
ARRY-797 Array BioPharma LMNA-related dilated cardiomyopathy Phase II
(p38 mitogen-activated Boulder, CO www.arraybiopharma.com
protein kinase inhibitor)
CAP-1002 Capricor Therapeutics Duchenne muscular dystrophy-related Phase I/II
(allogeneic cardiosphere-derived Beverly Hills, CA cardiomyopathy www.capricor.com
stem cell therapy) (see also heart attack)
MYK-461/SAR439152 MyoKardia symptomatic, obstructive hypertrophic Phase II
(myosin inhibitor) South San Francisco, CA cardiomyopathy www.myokardia.com
ORPHAN DRUG Sanofi
Bridgewater, NJ
non-obstructive hypertrophic Phase I
cardiomyopathy www.myokardia.com
MYK-491 MyoKardia dilated cardiomyopathy Phase I
(allosteric activator of myosin) South San Francisco, CA www.myokardia.com
Sanofi
Bridgewater, NJ
Cardiomyopathy
Drug Name Sponsor Indication Development Status
PB-1046 PhaseBio Pharmaceuticals Duchenne muscular dystrophy- Phase I
(VPAC2 selective agonist) Malvern, PA induced cardiomyopathy www.phasebio.com
ORPHAN DRUG (see also heart failure, pulmonary
vascular disease)
tafamidis meglumine Pfizer transthyretin amyloid cardiomyopathy Phase III
(amyloid inhibitor) New York, NY (Fast Track) www.pfizer.com
ORPHAN DRUG
Coronary Artery Disease
Drug Name Sponsor Indication Development Status
apabetalone Resverlogix secondary prevention of major Phase III
(BET protein inhibitor) San Francisco, CA adverse cardiac events (MACE) in www.resverlogix.com
patients with coronary artery disease,
type 2 diabetes and low HDL
cholesterol
AZD5718 AstraZeneca coronary artery disease Phase II
(5-lipoxygenase activating Wilmington, DE www.astrazeneca.com
protein inhibitor)
Brilinta® AstraZeneca cardiovascular outcomes in patients Phase III
ticagrelor Wilmington, DE with coronary artery disease and www.astrazeneca.com
type 2 diabetes without a previous
history of myocardial infarction
or stroke
Coronary Artery Disease
Drug Name Sponsor Indication Development Status
COMBO Stent™ OrbusNeich prevention of coronary artery in clinical trials
(anti-CD34 mAb/sirolimus drug Fort Lauderdale, FL restenosis in patients with coronary www.orbusneich.com
eluting stent) artery disease
CVBT-141H CardioVascular BioTherapeutics severe coronary heart disease Phase II
(fibroblast growth factor-1) Dallas, TX www.cvbt.com
DP9 Dimera myocardial ischemia Phase II completed
(progesterone transdermal) Portland, OR www.dimera.net
Generx Angionetics refractory angina due to myocardial Phase III
alferminogene tadenovec San Diego, CA ischemia (cardiac microvascular www.angionetics.com
(recombinant angiogenic insufficiency) (Fast Track)
gene therapy)
MEDI5884 MedImmune coronary heart disease Phase II
(cholesterol modulator) Gaithersburg, MD www.medimmune.com
MEDI6012 MedImmune coronary artery disease Phase II
(LCAT therapy) Gaithersburg, MD www.medimmune.com
ridaforolimus eluting coronary stent Medinol coronary artery restenosis Phase III
(device drug combination) Tel Aviv, Israel www.medinol.com
umirolimus Biosensors International coronary artery restenosis Phase III
(drug-eluting stent) Singapore www.biosensors.com
Coronary Artery Disease
Drug Name Sponsor Indication Development Status
VB-201 VBL Therapeutics atherosclerosis Phase II
Modi’in, Israel www.vblrx.com
Heart Attack
Drug Name Sponsor Indication Development Status
BVI-007 BioVascular prevention of myocardial infarction Phase I
(thrombopoietin inhibitor) San Diego, CA in patients who have had a previous www.biovascularinc.com
cardiovascular event
(see also stroke)
CAP-1002 Capricor Therapeutics myocardial infarction Phase I/II
(allogeneic cardiosphere-derived Beverly Hills, CA (see also cardiomyopathy) www.capricor.com
stem cell therapy)
CSL 112 CSL Behring reduction of recurrent cardiovascular Phase II
(apolipoprotein A-I infusion therapy) King of Prussia events following myocardial infarction www.cslbehring.com
Entresto® Novartis post-acute myocardial infarction Phase III
sacubitril/valsartan East Hanover, NJ (see also heart failure) www.novartis.com
FDY-5301 Faraday Pharmaceuticals myocardial infarction Phase II
Seattle, WA www.faradaypharma.com
Heart Attack
Drug Name Sponsor Indication Development Status
mesenchymal stem cell therapy Stemedica Cell Technologies acute myocardial infarction Phase I completed
(ischemic-tolerant stem cells) San Diego, CA (see also heart failure, stroke) www.stemedica.com
MultiStem® Athersys acute myocardial infarction Phase II
allogeneic stem cell therapy Cleveland, OH (see also stroke) www.athersys.com
REC-01 Recardio acute myocardial infarction Phase I
(CD26 antigen inhibitor) San Francisco, CA www.recardio.eu
RGN-352 injectable RegeneRx Biopharmaceuticals acute myocardial infarction Phase I completed
(thymosin beta-4) Rockville, MD (see also stroke) www.regenerx.com
TF0023 Techfields Pharma heart attack recovery Phase II
(aspirin pro-drug) Dover, DE (see also stroke) www.tfpharma.com
Heart Failure
Drug Name Sponsor Indication Development Status
AdipoCell™ U.S. Stem Cell congestive heart failure Phase I/II
adipose-derived autologous Miami, FL www.us-stemcell.com
stem cell therapy
Aironite Savara Pharmaceuticals diastolic heart failure Phase II
sodium nitrite inhalation Austin, TX www.savarapharma.com
Heart Failure
Drug Name Sponsor Indication Development Status
AMG 986 Amgen heart failure Phase I
(apelin receptor agonist) Thousand Oaks, CA www.amgen.com
AR-06 Arbor Pharmaceuticals heart failure Phase III
(hydralazine/isosorbide dinitrate Atlanta, GA www.arborpharma.com
controlled release)
AZD4831 AstraZeneca heart failure with a preserved Phase I
(myeloperoxidase modulator) Wilmington, DE ejection fraction www.astrazeneca.com
BAY1142524 Bayer HealthCare Pharmaceuticals left ventricular dysfunction Phase II
(chymase inhibitor) Whippany, NJ (heart failure) www.pharma.bayer.com
BMS-986224 Bristol-Myers Squibb chronic heart failure Phase I
Princeton, NJ www.bms.com
BMS-986231 Bristol-Myers Squibb acute heart failure Phase II
(nitroxyl donor) Princeton, NJ www.bms.com
BR6819 Bayer HealthCare Pharmaceuticals heart failure Phase I
(vasopressin receptor antagonist) Whippany, NJ www.pharma.bayer.com
CardiALLO ™ BioCardia heart failure Phase I
(allogenic culture expanded San Carlos, CA www.biocardia.com
mesenchymal stem cells derived
from bone marrow)
Heart Failure
Drug Name Sponsor Indication Development Status
CLR325 Novartis chronic stable heart failure Phase II
East Hanover, NJ www.novartis.com
elamipretide Stealth BioTherapeutics acute heart failure, heart failure with Phase II
Newton, MA preserved ejection fraction, heart www.stealthbt.com
failure with reduced ejection fraction
Entresto® Novartis heart failure with preserved Phase III
sacubitril/valsartan East Hanover, NJ ejection fraction www.novartis.com
(see also heart attack)
EZR104 Ezra Pharma heart failure application submitted
(valsartan extended release) Little Rock, AK (see also hypertension) www.ezrapharma.com
Farxiga® AstraZeneca worsening heart failure or cardio- Phase III
dapagliflozin Wilmington, DE vascular death in patients with www.astrazeneca.com
chronic heart failure
(see also other)
Furoscix scPharmaceuticals chronic heart failure application submitted
subcutaneous furosemide Burlington, MA www.scpharmaceuticals.com
decompensated heart failure Phase II/III
www.scpharmaceuticals.com
Heart Failure
Drug Name Sponsor Indication Development Status
GSK2798745 GlaxoSmithKline heart failure Phase II
(TRPV4 antagonist) Research Triangle Park, NC www.gsk.com
Injectafer® Daiichi Sankyo heart failure with iron deficiency and Phase III
ferric carboxymaltose Basking Ridge, NJ with a reduced ejection fraction www.dsi.com
Luitpold Pharmaceuticals
Shirley, NY
IW-1973 Ironwood Pharmaceuticals heart failure with preserved ejection Phase II
(soluble guanylate cyclase agonist) Cambridge, MA fraction www.ironwoodpharma.com
ixmyelocel-T Vericel heart failure due to ischemic Phase II completed
(stem cell therapy) Cambridge, MA dilated cardiomyopathy (Fast Track) www.vcel.com
ORPHAN DRUG
Jardiance® Boehringer Ingelheim heart failure with preserved ejection Phase III
empagliflozin Ridgefield, CT fraction www.boehringer-ingelheim.com
Eli Lilly
Indianapolis, IN
JVS-100 Juventas Therapeutics heart failure (Fast Track) Phase II
(non-viral gene therapy Cleveland, OH (see also peripheral vascular disease) www.juventasinc.com
expressing SDF-1)
LIK066 Novartis patients with type 2 diabetes and Phase II
(SGLT 1/2 inhibitor) East Hanover, NJ heart failure www.novartis.com
Heart Failure
Drug Name Sponsor Indication Development Status
long-acting loop diuretic Sarfez Pharmaceuticals chronic congestive heart failure Phase II
Vienna, VA www.sarfezpharma.com
mesenchymal stem cell therapy Stemedica Cell Technologies chronic heart failure Phase II
(ischemic-tolerant stem cells) San Diego, CA (see also heart attack, stroke) www.stemedica.com
MPC-150-IM Mesoblast advanced heart failure Phase III
(human mesenchymal stem New York, NY www.mesoblast.com
cell therapy)
end-stage heart failure Phase II
www.mesoblast.com
MyoCell® U.S. Stem Cell severe heart damage in heart failure Phase II/III
muscle-derived autologous Sunrise, FL www.us-stemcell.com
stem cell therapy
neladenoson bialanate Bayer HealthCare Pharmaceuticals chronic heart failure Phase II
(adenosine A1 receptor agonist) Whippany, NJ www.pharma.bayer.com
Neucardin Zensun USA chronic heart failure Phase II
recombinant human neuregulin-1 beta San Diego, CA www.zensunus.com
omecamtiv mecarbil Cytokinetics heart failure Phase III
(cardiac myosin activator) South San Francisco, CA www.amgen.com
Amgen
Thousand Oaks, CA
Heart Failure
Drug Name Sponsor Indication Development Status
Opsumit® Actelion Pharmaceuticals heart failure with preserved ejection Phase II
macitentan South San Francisco, CA fraction and pulmonary arterial www.actelion.com
disease
(see also pulmonary vascular disease,
other)
PB-1046 PhaseBio Pharmaceuticals heart failure with preserved or reduced Phase II
(VPAC2 selective agonist) Malvern, PA ejection fraction www.phasebio.com
(see also cardiomyopathy,
pulmonary vascualr disease)
PL-3994 NPR-A Palatin Technologies heart failure Phase II
(cyclic natriuretic peptide Cranbury, NJ www.palatin.com
receptor A agonist)
REC-02 Recardio congestive heart failure Phase I
(chemokine CXCL12 modulator) San Francisco, CA www.recardio.eu
RT-100 Renova Therapeutics congestive heart failure (Fast Track) Phase III
(gene therapy) San Diego, CA www.renovatherapeutics.com
RT-400 Renova Therapeutics acute decompensated heart failure Phase II
(peptide therapy) San Diego, CA www.renovatherapeutics.com
SAR440181 Sanofi post acute heart failure Phase I
(DCM1 myosin activation) Bridgewater, NJ www.sanofi.com
Heart Failure
Drug Name Sponsor Indication Development Status
SERCA 2a gene therapy Theragene Pharmaceuticals NYHA class III/IV heart failure Phase II
San Diego, CA (Fast Track) www.theragenepharma.com
TD-0714 Theravance Biopharma heart failure Phase I
(neprilysin inhibitor) South San Francisco, CA www.theravance.com
TD-1439 Theravance Biopharma heart failure Phase I
(neprilysin inhibitor) South San Francisco, CA www.theravance.com
ularitide Cardiorentis acute heart failure (Fast Track) Phase III
(atrial peptide agonist/diuretic) Zug, Switzerland www.cardiorentis.com
vericiguat Bayer HealthCare Pharmaceuticals chronic heart failure Phase III
(sGC stimulator) Whippany, NJ www.pharma.bayer.com
Hypertension
Drug Name Sponsor Indication Development Status
AF-130 Merck hypertension Phase I completed
(purinergic P2X3 receptor Kenilworth, NJ www.merck.com
antagonist)
aprocitentan Idorsia Pharmaceuticals resistant hypertension management Phase II
(dual endothelin receptor Allschwil, Switzerland www.adorsia.com
antagonist) Janssen Biotech www.janssen.com
Horsham, PA
Hypertension
Drug Name Sponsor Indication Development Status
atorvastatin/aspirin/ramipril fixed- Ferrer Internacional hypertension Phase II
dose combination (polypill) Barcelona, Spain www.ferrer.com
B244 AOBiome hypertension Phase II
(bacteria-based therapeutic) Cambridge, MA www.aobiome.com
digoxin immune Fab AMAG Pharmaceuticals severe preeclampsia Phase II/III
ORPHAN DRUG Waltham, MA (Fast Track) www.amagpharma.com
Edarbi® Arbor Pharmaceuticals hypertension (pediatric) Phase III
azilsartan medoxomil Atlanta, GA www.arborpharma.com
EZR104 Ezra Pharma hypertension application submitted
(valsartan extended release) Little Rock, AK (see also heart failure) www.ezrapharma.com
IONIS-AGT-LRX Ionis Pharmaceuticals treatment resistant hypertension Phase II
(LICA-conjugated antisense drug) Carlsbad, CA www.ionispharma.com
KBP-5074 KBP Biosciences hypertension in advanced chronic Phase I/II
(mineralocorticoid receptor antagonist) Princeton, NJ kidney disease www.kbpbiosciences.com
KIT-302 Kitov Pharmaceuticals treatment of hypertension and application submitted
(amlodipine/celecoxib fixed-dose Tel Aviv, Israel osteoarthritis pain simultaneously www.kitovpharma.com
combination)
Hypertension
Drug Name Sponsor Indication Development Status
Kybiqo Stocosil hypertension and hyperlipidemia Phase II
olmesartan medoxomil/rosuvastatin Industry, CA (see also lipid disorders) www.stocosil.com
fixed-dose combination
LHW090 Novartis resistant hypertension Phase II
East Hanover, NJ www.novartis.com
QGC001 Quantum Genomics hypertension Phase II
(brain aminopeptidase A inhibitor) Paris, France www.quantum-genomics.com
RMJH-111b RMJ Holdings essential hypertension Phase I/II completed
(magnesium citrate) Ashburn, VA
Lipid Disorders
Drug Name Sponsor Indication Development Status
1-MNA Cortria hyperlipidemia Phase II completed
(active metabolite of niacin) Boston, MA www.pharmena.eu
Pharmena
Lodz, Poland
AKCEA-ANGPTL3-LRX Akcea Therapeutics rare hyperlipidemias Phase II
(ANGPTL3 protein inhibitor) Cambridge, MA www.akceatx.com
AKCEA-APO(a)-LRX Akcea Therapeutics hyperlipoproteinemia with Phase II
(ligand conjugated antisense drug) Cambridge, MA significant cardiovascular risk www.akceatx.com
Lipid Disorders
Drug Name Sponsor Indication Development Status
AKCEA-APOCIII-LRX Akcea Therapeutics hypertriglyceridemia Phase II
(ligand conjugated antisense drug) Cambridge, MA www.akceatx.com
AMG 899 Amgen dyslipidemia Phase II
(CETP inhibitor) Thousand Oaks, CA www.amgen.com
ARI-3037MO Arisaph Pharmaceuticals dyslipidemia, hypertriglyceridemia Phase II
(GPR109A/GPR109B receptor agonist) Boston, MA www.arisaph.com
bempedoic acid (oral) Esperion Therapeutics lowering elevated low-density Phase III
Ann Arbor, MI lipoprotein (LDL) cholesterol, www.esperion.com
cardiovascular disease risk reduction
bempedoic acid/ezetimibe Esperion Therapeutics lowering elevated LDL cholesterol Phase III
fixed-dose combination (oral) Ann Arbor, MI www.esperion.com
CaPre® Acasti Pharma hypertriglyceridemia Phase II completed
long-chain Omega-3 phospholid Quebec, Canada www.ascatipharma.com
Epanova® AstraZeneca high-risk for cardiovascular events Phase III
omega-3 carboxylic acids Wilmington, DE in patients with persistent hypertrigly- www.astrazeneca.com
ceridemia and low HDL cholesterol
evinacumab (REGN1500) Regeneron homozygous familial Phase II
(ANGPTL-1 antibody) Tarrytown, NY hypercholesterolemia www.regeneron.com
Lipid Disorders
Drug Name Sponsor Indication Development Status
gemcabene Gemphire Therapeutics severe hypertriglyceridemia, Phase II
(acetyl-CoA carboxylase inhibitor/ Livonia, MI homozygous familial www.gemphire.com
lipoprotein A inhibitor) hypercholesterolemia, heterozygous
ORPHAN DRUG familial hypercholesterolemia
inclisiran The Medicines Company hypercholesterolemia Phase III
(RNAi therapeutic) Parsippany, NJ www.themedicinescompany.com
K-312 Kowa Research Institute dyslipidemia Phase I
Morrisville, NC www.kowaus.com
Kybiqo Stocosil hypertension and hyperlipidemia Phase II
olmesartan medoxomil/rosuvastatin Industry, CA (see also hypertension) www.stocosil.com
fixed-dose combination
Kynamro® Kastle Therapeutics heterozygous familial Phase II completed
mipomersen Chicago, IL hypercholesterolemia www.kastletherapeutics.com
MGL-3196 Madrigal Pharmaceuticals heterozygous familial Phase II
(liver-directed thyroid hormone West Conshohocken, PA hypercholesterolemia www.madrigalpharma.com
receptor beta agonist)
pemafibrate (K-877) Kowa Research Institute dyslipidemia Phase III
(selective PPAR-alpha modulator) Morrisville, NC www.kowaus.com
PF-06427878 Pfizer hyperlipidemia Phase I
(DGAT2 inhibitor) New York, NY www.pfizer.com
Lipid Disorders
Drug Name Sponsor Indication Development Status
Praluent® Regeneron homozygous familial Phase III
alirocumab Tarrytown, NY hypercholesterolemia www.regeneron.com
ORPHAN DRUG Sanofi (see also acute coronary syndrome) www.sanofi.com
Bridgewater, NJ
RGX 501 REGENXBIO homozygous familial Phase I/II
(personalized gene therapy) Rockville, MD hypercholesterolemia www.regenxbio.com
ORPHAN DRUG University of Pennsylvania
Philadelphia, PA
saroglitazar Zydus Cadila dyslipidemia (≥500 mg/dL) Phase II
Gujarat, India www.zyduscadila.com
SC401 Sancilio severe hypertriglyceridemia Phase II
(docosahexaenoic acid/ Riveria Beach, FL www.sancilio.com
eicosapentaenoic acid)
VK-2809 Viking Therapeutics hypercholesterolemia Phase II
(thyroid hormone receptor San Diego, CA www.vikingtherapeutics.com
beta agonist)
volanesoren Akcea Therapeutics familial chylomicroenemia application submitted
(antisense ApoCIII inhibitor) Cambridge, MA syndrome www.akceatx.com
ORPHAN DRUG
Peripheral Vascular Disease
Drug Name Sponsor Indication Development Status
ACP-01 Hemostemix critical limb ischemia Phase II
(angiogenic cell precursor therapy) Calgary, Canada www.hemostemix.com
ALD-301 Aldagen peripheral arterial occlusive disorders Phase II completed
(bone marrow-derived adult stem cells) Durham, NC
ASCT01 Lifecells critical limb ischemia Phase I/II
(autologous stem cell therapy) Irvine, CA www.lifecellsllc.com
autologous stem cell therapy BioTech Holdings ischemia associated with diabetes in clinical trials
Richmond, CA
BAY1193397 Bayer HealthCare Pharmaceuticals peripheral arterial disease Phase II
(AR alpha-2c receptor antagonist) Whippany, NJ www.pharma.bayer.com
beperminogene perplasmid AnGes USA critical limb ischemia Phase III
(HGF plasmid) Bethesda, MD (Fast Track) www.anges.co.jp
Mitsubishi Tanabe Pharma
Osaka, Japan
CVBT-141C CardioVascular BioTherapeutics peripheral artery disease Phase II
(fibroblast growth factor-1) Dallas, TX www.cvbt.com
human plasma-derived fibrinolysin Grifols Therapeutics acute peripheral arterial occlusion Phase II
ORPHAN DRUG Los Angeles, CA www.grifols.com
Peripheral Vascular Disease
Drug Name Sponsor Indication Development Status
JVS-100 Juventas Therapeutics peripheral artery disease (Fast Track) Phase II
(non-viral gene therapy) Cleveland, OH (see heart failure) www.juventasinc.com
Liprostin™ AngioSoma peripheral artery disease Phase II
alprostadil (liposomal Montgomery, TX www.antisomainfo
controlled release)
LLG783 Novartis intermittent claudication, Phase II
(LDL receptor antagonist mAb) East Hanover, NJ peripheral arterial disorder www.novartis.com
MABp1 XBiotech peripheral vascular disease (Fast Track) Phase II
(IL-1-alpha/IgG1 mAb) Austin, TX www.xbiotech.com
MultiGeneAngio MultiGene Vascular Systems peripheral arterial disease Phase I/II
peripheral arterial disease cell therapy Haifa, Israel www.mgvs.co.il
NVS therapy Alucent Medical peripheral vascular disease Phase I
(peripheral vascular disorder Salt Lake City, UT www.alucentmedical.com
drug-device therapy)
PLX-PAD cell therapy Pluristem Therapeutics critical limb ischemia (Fast Track) Phase III
Haifa, Israel www.pluristem.com
SB-030 Symic Bio peripheral arterial disease Phase II
(inflammation mediator modulator) Emeryville, CA www.symic.bio
Peripheral Vascular Disease
Drug Name Sponsor Indication Development Status
TV1001SR Theravasc peripheral arterial disease Phase II
(sodium nitrate oral) Cleveland, OH www.theravasc.com
VM202 ViroMed peripheral artery disease Phase III
(donaperminogene seltoplasmid) Seoul, South Korea www.viromed.co.kr
vonapanitase Proteon Therapeutics peripheral artery disease Phase III
(recombinant human elastase) Waltham, MA (Fast Track) www.proteontherapeutics.com
ORPHAN DRUG
Xarelto® Bayer HealthCare Pharmaceuticals infrainguinal peripheral arterial Phase III
rivaroxaban Whippany, NJ disease www.pharma.bayer.com
Janssen (see also stroke, thrombosis, other) www.janssen.com
Raritan, NJ
Pulmonary Vascular Disease
Drug Name Sponsor Indication Development Status
ABI-009 Aadi pulmonary arterial hypertension Phase I
(sirolimus albumin bound Pacific Palisades, CA
nanoparticle)
ambrisentan/tadalafil GlaxoSmithKline pulmonary arterial hypertension Phase I completed
fixed-dose combination Research Triangle Park, NC www.gsk.com
Pulmonary Vascular Disease
Drug Name Sponsor Indication Development Status
bardoxolone methyl Reata Pharmaceuticals connective tissue disease associated Phase III
(Nrf2 activator) Irving, TX with pulmonary arterial hypertension www.reatapharma.com
ORPHAN DRUG (CTD-PAD)
pulmonary hypertension-interstitial Phase II
lung disease (PH-ILD) www.reatapharma.com
beraprost 314d Lung Biotechnology pulmonary arterial hypertension Phase III
Silver Spring, MD www.lungbiotechnology.com
CAM2043 Camurus pulmonary arterial hypertension Phase I/II
(treprostinil controlled release) Lund, Sweden www.camurus.com
GeNOsyl GeNO pulmonary arterial hypertension Phase II completed
nitric oxide inhalation Atlanta, GA www.genollc.com
GSK2256098 GlaxoSmithKline pulmonary arterial hypertension Phase I completed
(FAK inhibitor) Research Triangle Park, NC www.gsk.com
GSK2586881 GlaxoSmithKline pulmonary arterial hypertension Phase I completed
(recombinant human angiotensin Research Triangle Park, NC www.gsk.com
converting enzyme)
INOpulse Bellerophon Therapeutics pulmonary arterial hypertension Phase III
nitric oxide inhalation Warren, NJ www.bellerophon.com
ORPHAN DRUG
Pulmonary Vascular Disease
Drug Name Sponsor Indication Development Status
INS1009 Insmed pulmonary arterial hypertension Phase I
(inhaled, sustained-release Bridgewater, NJ www.insmed.com
nebulized prodrug formulation
of treprostinil)
KAR5585 Karos Pharmaceuticals pulmonary arterial hypertension Phase I
(tryptophan hydroxylase 1 inhibitor) New Haven, CT
ORPHAN DRUG
Letairis® Gilead Sciences pulmonary arterial hypertension Phase II
ambrisentan Foster City, CA (pediatric) www.gilead.com
GlaxoSmithKline www.gsk.com
Research Triangle Park, NC
levosimendan IV Tenax Therapeutics pulmonary hypertension due to heart Phase II
(R-enantiomer of simendan) Morrisville, NC failure with preserved ejection fraction www.tenaxthera.com
(see also adjunctive therapies)
LIQ861 Liquidia Technologies pulmonary arterial hypertension Phase III
(inhalation) Morrisville, NC www.liquidia.com
Opsumit® Actelion Pharmaceuticals pulmonary arterial hypertension Phase III
macitentan South San Francisco, CA (pediatric), portopulmonary www.actelion.com
ORPHAN DRUG hypertension
(see also heart failure, other)
chronic thromboembolic pulmonary Phase II
hypertension www.actelion.com
Pulmonary Vascular Disease
Drug Name Sponsor Indication Development Status
OreniLeft™ United Therapeutics pulmonary arterial hypertension Phase III
treprostinil Silver Spring, MD associated with left ventricular www.unither.com
diastolic dysfunction (WHO group 2)
PB-1046 PhaseBio Pharmaceuticals pulmonary arterial hypertension Phase II
(VPAC2 selective agonist) Malvern, PA (see also cardiomyopathy, www.phasebio.com
ORPHAN DRUG heart failure)
ralinepag Arena Pharmaceuticals pulmonary arterial hypertension Phase II
(prostacyclin receptor agonist) San Diego, CA www.arenapharm.com
ORPHAN DRUG
RemoSynch™ United Therapeutics pulmonary arterial hypertension application submitted
implantable system for Remodulin® Silver Spring, MD www.unither.com
(treprostinil)
RemUnity™ United Therapeutics pulmonary arterial hypertension Phase III
treprostinil continuous subcutaneous Silver Spring, MD www.unither.com
via pre-filled pump
sGC activator 2 Bayer HealthCare Pharmaceuticals pulmonary hypertension Phase I
(soluble guanylate cyclase) Whippany, NJ www.pharma.bayer.com
sildenafil Pfizer persistent pulmonary arterial Phase III
New York, NY hypertension (neonates) www.pfizer.com
Pulmonary Vascular Disease
Drug Name Sponsor Indication Development Status
tacrolimus VIVUS pulmonary arterial hypertension Phase II
ORPHAN DRUG Campbell, CA www.vivus.com
Trevyent SteadyMed Therapeutics pulmonary arterial hypertension application submitted
treprostinil patch pump San Ramon, CA www,steadymed.com
ORPHAN DRUG
Tysuberprost™ United Therapeutics pulmonary arterial hypertension Phase III
esuberaprost in combination Silver Spring, MD (decrease morbidity and mortality) www.unither.com
with Tyvaso® (treprostinil)
Tyvaso® United Therapeutics pulmonary arterial hypertension Phase III
treprostinil Silver Spring, MD associated with chronic obstructive www.unither.com
pulmonary disease (WHO group 3)
Tyvaso-ILD™ United Therapeutics pulmonary arterial hypertension Phase III
treprostinil Silver Spring, MD associated with idiopathic www.unither.com
pulmonary fibrosis (WHO group 3)
Uptravi® Actelion Pharmaceuticals pulmonary arterial hypertension Phase III
selexipag intravenous South San Francisco, CA (switch from oral to intravenous) www.actelion.com
VentaProst Aerogen Pharma pulmonary hypertension Phase II
epoprostenol inhalation Galway, Ireland www.areogen.com
Stroke
Drug Name Sponsor Indication Development Status
3K3A-APC ZZ Biotech moderate to severe ischemic stroke Phase II
(recombinant human wild-type Houston, TX www.zzbiotech.com
activated protein C)
BIIB093 (glibenclamide IV) Biogen large hemispheric infarction Phase II
ORPHAN DRUG Cambridge, MA (severe form of ischemic stroke) www.biogen.com
(Fast Track)
BVI-007 BioVascular prevention of thrombotic stroke Phase I
(thrombopoietin inhibitor) San Diego, CA in patients who have had a previous www.biovascularinc.com
cardiovascular event
(see also heart attack)
clazosentan Idorsia Pharmaceuticals cerebral vasospasm associated with Phase III
(endothelin receptor antagonist) Allschwil, Switzerland aneurysmal subarachnoid hemorrhage www.idorsia.com
ORPHAN DRUG
CN-105 AegisCN intracerebral hemorrhage Phase II
(apolipoprotein E agonist) Raleigh, NC
DM199 DiaMedica Therapeutics acute ischemic stroke Phase II
(recombinant human kallikrein-1) Minneapolis, MN www.diamedica.com
DS-1040 Daiichi Sankyo acute ischemic stroke Phase I
(TAFIα inhibitor) Basking Ridge, NJ www.dsi.com
Stroke
Drug Name Sponsor Indication Development Status
EG-1962 Edge Therapeutics aneurysmal subarachnoid hemorrhage Phase III
(nimodipine microparticles) Berkeley Heights, NJ (intraventricular delivery) (Fast Track) www.edgetherapeutics.com
ORPHAN DRUG
aneurysmal subarachnoid hemorrhage Phase I
(intracisternal delivery) www.edgetherapeutics.com
LT-3001 Lumosa Therapeutics acute ischemic stroke Phase I
(antioxidant/free radical scavenger) Taipei, Taiwan www.lumosa.com.tw
MAA868 Novartis stroke prevention in atrial fibrillation Phase II
(anti-FXI mAb) East Hanover, NJ www.novartis.com
mesenchymal stem cell therapy Stemedica Cell Technologies ischemic stroke Phase II
(ischemic-tolerant stem cells) San Diego, CA (see also heart attack, heart failure) www.stemedica.com
MultiStem® Athersys ischemic stroke (Fast Track) Phase II
allogeneic stem cell therapy Cleveland, OH (see also heart attack) www.athersys.com
NA-1 NoNO stroke with endovascular intervention Phase III
(PSD-95 antagonist) Toronto, Canada www.nonoinc.ca
natalizumab Biogen acute ischemic stroke Phase II
(alpha-4 integrin inhibitor) Cambridge, MA www.biogen.com
Stroke
Drug Name Sponsor Indication Development Status
NVX-208 NuvOx Pharma acute ischemic stroke Phase I/II
(dodecafluoropentane nanoemulsion) Tucson, AZ www.nuvoxpharma.com
PF-05230907 Pfizer intracerebral hemorrhage Phase I
(factor Xa protein replacement) New York, NY www.pfizer.com
ORPHAN DRUG
Pradaxa® Boehringer Ingelheim Pharmaceuticals secondary stroke prevention in Phase III
dabigatran etexilate Ridgefield, CT patients with embolic stroke of www.boehringer-ingelheim.com
undetermined source
(see also thrombosis)
RGN-352 injectable RegeneRx Biopharmaceuticals stroke Phase I completed
(thymosin beta-4) Rockville, MD (see also heart attack) www.regenerx.com
salvinorin A Neurokappa Therapeutics cerebral vasospasm Phase I
(opioid kappa receptor agonist) Philadelphia, PA
SB623 SanBio ischemic stroke Phase II
(modified bone marrow derived Mountain View, CA www.sanbio.com
mesenchymal stem cells) Sunovion Pharmaceuticals www.sunovion.com
Marlborough, MA
stroke polyfunctional therapeutic HSRx Biopharmaceutical stroke Phase I
Tucson, AZ www.hsrxbiopharmaceutical.com
Stroke
Drug Name Sponsor Indication Development Status
TF0023 Techfields Pharma ischemic stroke Phase II
(aspirin pro-drug) Dover, DE (see also heart attack) www.tfpharma.com
Xarelto® Bayer HealthCare Pharmaceuticals secondary prevention of stroke in Phase III
rivaroxaban Whippany, NJ patients who have had an embolic www.pharma.bayer.com
Janssen stroke www.janssen.com
Raritan, NJ (see also peripheral vascular disease,
thrombosis, other)
Thrombosis
Drug Name Sponsor Indication Development Status
BAY1213790 Bayer HealthCare Pharmaceuticals thrombosis Phase II
(anti-FXIa antibody) Whippany, NJ www.pharma.bayer.com
BAY2306001 Bayer HealthCare Pharmaceuticals thrombosis Phase II
(FXI antisense drug/IONIS-FXI) Whippany, NJ www.pharma.bayer.com
Ionis Pharmaceuticals
Carlsbad, CA
BMS-986177 Bristol-Myers Squibb thrombosis Phase I
(fibrinolytic agent) Princeton, NJ www.bms.com
Thrombosis
Drug Name Sponsor Indication Development Status
Eliquis® Bristol-Myers Squibb acute venous thromboembolism (VTE) Phase III
apixaban Princeton, NJ (pediatric) www.bms.com
Pfizer www.pfizer.com
New York, NY
thromboembolism (pediatric) Phase I
www.bms.com
www.pfizer.com
EP-7041 eXIthera Pharmaceuticals prevention and treatment of Phase I
(factor XIa inhibitor) Westborough, MA thrombosis in patients undergoing www.exithera.com
surgeries
ichorcumab Janssen thrombosis Phase I
(recombinant human anti-thrombin Raritan, NJ www.janssen.com
antibody)
isoquercetin Quercegen Pharmaceutical prevention of thromboembolism Phase II/III
(plant flavinoid) Sudbury, MA in cancer patients www.quercegen.com
NOV-12 Morphosys prevention of thrombosis Phase I
Munich, Germany www.morphosys.com
Novartis www.novartis.com
East Hanover, NJ
Pradaxa® Boehringer Ingelheim VTE (pediatric) Phase III
dabigatran etexilate Ridgefield, CT (see also stroke) www.boehringer-ingelheim.com
Thrombosis
Drug Name Sponsor Indication Development Status
tecarfarin Espero Biopharma thrombosis Phase III
Jacksonville, FL www.esperobio.com
TS23 Translational Sciences thrombosis Phase I
(alpha-2 plasmin inhibitor Phoenix, AZ www.translationalsciences.com
inactivating mAb)
warfarin breakable tablets Accu-Break Pharmaceuticals thromboembolism application submitted
Plantation, FL
Xarelto® Janssen prevention of symptomatic VTE and Phase III
rivaroxaban Raritan, NJ VTE-related death in high-risk, www.janssen.com
medically ill patients
(see also peripheral vascular disease,
stroke, other)
VTE prevention in high-risk ambulatory Phase III
cancer patients www.janssen.com
long-term prevention of recurrent Phase III
symptomatic VTE www.janssen.com
Other
Drug Name Sponsor Indication Development Status
AMG 529 Amgen cardiovascular disorders Phase I
(asialoglycoprotein receptor 1 Thousand Oaks, CA www.amgen.com
inhibitor)
AZD8601 AstraZeneca cardiovascular disorders Phase II
(VEGF-A) Wilmington, DE www.astrazeneca.com
canakinumab (ACZ885) Novartis secondary prevention of cardiovascular Phase III
(anti-IL1 beta mAb) East Hanover, NJ events www.novartis.com
COR-001 Corvidia Therapeutics cardiovascular disorders Phase I
(monoclonal antibody) Waltham, MA www.corvidiatx.com
Farxiga® AstraZeneca renal outcomes and cardiovascular Phase III
dapagliflozin Wilmington, DE death in patients with chronic www.astrazeneca.com
kidney disease
(see also heart failure)
cardiovascular outcomes in patients Phase III
with type 2 diabetes www.astrazeneca.com
FP-045 Foresee Pharmaceuticals cardiovascular disorders Phase I
(aldehyde dehydrogenase 2 agonist) Newark, DE www.foreseepharma.com
Other
Drug Name Sponsor Indication Development Status
Opsumit® Actelion Pharmaceuticals in patients with congenital heart Phase III
macitentan South San Francisco, CA defects who underwent Fontan-palliation www.actelion.com
(see also heart failure, pulmonary
vascular disease)
SAR247799 Sanofi cardiovascular disorders Phase I
(S1P1 agonist) Bridgewater, NJ www.sanofi.com
SAR407899 Sanofi microvascular angina Phase II
(rho kinase) Bridgewater, NJ (Syndrome X) www.sanofi.com
SNF472 Sanifit reduce cardiovascular events in Phase II
(hexasodium phytate) San Diego, CA patients undergoing hemodialysis www.sanifit.com
sotagliflozin Sanofi reduce cardiovascular events in Phase III
Bridgewater, NJ high risk patients with type 2 www.sanofi.com
diabetes
T89 Tasly Pharmaceuticals chronic stable angina pectoris Phase III
Rockville, MD www.taslyus.com
TD-9855 Theravance Biopharma orthostatic hypotension Phase I
(monoamine reuptake inhibitor) South San Francisco, CA www.theravance.com
udenafil Mezzion Pharma single ventricle congenital heart disease Phase III
(type 5 cyclic nucleotide Seoul, South Korea (adolescents) www.mezzion.co.kr
phosphodiesterase inhibitor)
Other
Drug Name Sponsor Indication Development Status
Xarelto® Bayer HealthCare Pharmaceuticals reduce risk of MACE in patients with application submitted
rivaroxaban Whippany, NJ coronary artery disease or peripheral www.pharma.bayer.com
Janssen artery disease www.janssen.com
Raritan, NJ (see also peripheral vascular disease,
stroke, thrombosis)
reduce risk of MACE in patients with Phase III
congestive heart failure and significant www.pharma.bayer.com
coronary artery disease www.janssen.com
The content of this report has been obtained through public, government and industry sources, and the Springer "Adis Insight” database based on the latest
information. Report current as of January 24, 2018. The medicines in this listing include medicines being developed by U.S.-based companies conducting trials
in the United States abroad, PhRMA-member companies conducting trials in the United States and abroad, and foreign companies conducting clinical trials in
the United States. Some products may not be in active clinical trials. The information may not be comprehensive. For more, specific information about a particular
product, contact the individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development is available on PhRMA's
website: www.phrma.org.
Definitions
Application Submitted—An application for marketing has been submitted by the company to the U.S. Food and Drug Administration (FDA).
Fast Track—Upon request by a sponsor, the FDA can grant this designation to facilitate the development and expedite the review of a drug or biologic to treat a
serious condition and fill an unmet medical need. When considering a biopharmaceutical company’s request for Fast Track designation for an investigational
drug or biologic, the FDA evaluates whether it will affect factors such as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will
progress from a less severe condition to a more serious one, and whether a condition can be adequately addressed by available therapy. With Fast Track designation,
early and frequent communication between the FDA and the biopharmaceutical company is encouraged throughout the entire drug development and review
process to help to quickly resolve any questions or issues that arise, potentially leading to an earlier approval and access by patients.
Orphan Designation—Upon request by a sponsor, the FDA can grant special status (“orphan status”) to a drug or biologic to treat a rare disease or condition.
In order to receive an orphan designation, a qualifying drug or biologic must be intended for the treatment, diagnosis, or prevention of a rare disease or
condition that affects usually fewer than 200,000 people in the United States.
Phase I—Researchers test the investigational drug or biologic in a small group of people, usually between 20 and 100 healthy adult volunteers, to evaluate
its initial safety and tolerability profile, determine a safe dosage range, and identify potential side effects.
Phase II—The investigational drug or biologic is given to volunteer patients, usually between 100 and 500, to determine whether it is effective, identify an
optimal dose, and to further evaluate its short-term safety.
Phase III—The investigational drug or biologic is given to a larger, more diverse patient population, often involving between 1,000 and 5,000 patients
(but sometimes many more), to generate statistically significant evidence to confirm its safety and effectiveness. Phase III studies are the longest studies
and usually take place in multiple sites around the world.